Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study


This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML), chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood stem cell transplantation. * GVHD is a condition in which cells from the donor's tissue attack the organs. * RGI-2001 is an investigational treatment
SparkCures ID 1633
Trial Phase Phase 1
Enrollment 25 Patients
Trial Sponsors
  • Massachusetts General Hospital
NCT Identifier


Am I Eligible?

The following criteria is a partial list of reasons why patients may or may not be eligible to participate in this clinical trial. Further evaluation with a medical professional will be required to determine full eligibility.

The following criteria is provided for health care professionals.

Inclusion Criteria:

* Men or women ≥ 18 and ≤ 80 years old
* Diagnosis of hematological malignancy:

* Acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in morphologic complete remission
* Myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), or chronic myelomonocytic leukemic (CMML) with \< 5% blasts in blood or bone marrow
* Chemosensitive Hodgkin lymphoma (HL) or Non-Hodgkin lymphoma (NHL)
* Patients must be undergoing haploidentical allogeneic hematopoietic cell transplantation, defined as 1st or 2nd degree relative with at least 5/10 matching at HLA-A, -B, -C, DR, and DQ.
* ECOG performance status ≤2
* Patients with adequate physical function as measured by:

* Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction \>25%
* Hepatic:

* Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or hemolysis
* ALT, AST, and Alkaline Phosphatase \< 5 x ULN
* Renal: Serum creatinine within normal range, or if serum creatinine is outside normal range, then renal function (measured or estimated creatinine clearance or GFR) ≥ 40mL/min/1.73m2
* Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted
* Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

* Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received a prior autologous hematopoietic stem cell transplant.)
* Participants who are receiving any other investigational agents within 14 days prior to RGI-2001 dosing. Thus, participants must stop investigational agents by Day -9 prior to transplant.
* Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, recent myocardial infarction or stroke, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
* Patients with active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy.
* Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.
* Pregnant and breast-feeding women are ineligible because they are not eligible for hematopoietic stem cell transplantation.
* HIV-positive participants and patients with active Hepatitis B or C are ineligible

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